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  Street address:
Level 11, KGV Building
Missenden Road
Postal address:
Post Office Box M30
Missenden Road NSW 2050

Phone: (02) 9515-6111
Fax: (02) 9515-9610

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Cell and Molecular Therapies
Cell and Molecular Therapies

Cell and Molecular Therapies

Our Research

Revolutionary technologies using human cells and genetic engineering for the development of novel therapies could provide cures for many human diseases - heart disease, organ failure and some cancers, as well as genetic diseases such as haemophilia and thalassaemia. Our aim is to provide cellular therapies, regenerative medicines and tissue engineering based on a firm foundation of scientific evidence. We build on genetic technologies and disease models to understand the biology of adult stem cells and the causes of diseases including blood cancers, and genetic disorders.

Providing services to academic and industry partners for development, manufacture, clinical evaluation and implementation of cell and gene therapies.

Taking discovery research into clinical practice with robust cell manufacturing protocols to meet Therapeutic Goods Administration (TGA) requirements for biologicals.


Clinical grade processing

  • Haematopoietic progenitor cell (HPC) apheresis
  • Bone marrow harvest (BM)
  • Leukapheresis of mononuclear cells (MNC)
  • Isolation and expansion of Mesenchymal Stromal Cells (MSC)
  • Antigen presenting cells including dendritic cells (DC)
  • Tissue specific cells for regenerative medicine applications
  • Cell processing
  • Cell expansion in static culture and bioreactors
  • Cryopreservation
  • Gene modification of cells using retroviral, lentiviral and other viral vectors
  • Cell product characterisation by flow cytometry and PCR
  • Pharmaceutical formulation of viral vectors
  • Autologous serum eye drops

Conduct of clinical trials

  • Clinical trials - Phase I, II, III
  • Regulatory submissions to local and national bodies
  • Industry and Investigator initiated studies
  • Work to your IND or EU approved protocol
  • Fully compliant with ICH GCP standards
  • Experienced staff manage Study start-up to Close out
  • Long-term follow-up available

Clinical expertise

  • Cell - delivered gene therapies (thalassemia, haematological malignancies)
  • Viral vector gene therapies (haemophilia, hepatocellular carcinoma)
  • Immunotherapies (cancer, melanoma)


  • Provide compliant manufacturing and clinical trial services to academic and industry partners.
  • Support development, manufacture, clinical evaluation and implementation of novel cell and gene therapies.
  • Provide improved treatment options for patients.
  • Educate and train clinical, scientific and regulatory staff.

Clinical trials

Our Clinical Trials team offers patients the opportunity of access to novel cell and genetic therapies, often when little to no alternative therapeutic options may be available. Our aim is to allow people to live longer, healthier lives and reduce hospital visits by helping develop new treatments. Utilising the services of our integrated cell processing laboratory allows us the opportunity to collect, manage, store, prepare and dispense investigational cell and gene therapy products. In addition to running our own Clinical Trials we also support Pharmacy and other trials at Royal Prince Alfred Hospital with specialised storage of fully traceable patient specific products.


Cell and Molecular Therapies (CMT) is located at one of Australia's oldest tertiary referral public healthcare institutions with an outstanding record in clinical research and early adoption of novel technologies. We are adjacent to Australia's first tertiary education institution, the University of Sydney, ranked in the top 100 universities in the world.

The team led by Professor John Rasko has extensive cell and gene therapy expertise across all stages of product development. CMT focuses on product quality, through a combination of technical skills, project management, commitment to quality systems and operational excellence. CMT are committed to supporting the high quality research of collaborators in NSW, nationally and overseas. Manufacturing and research supported by CMT is performance driven to measurable outcomes.

Quality management

Operations are conducted in accordance with international, national, local and facility guidelines and regulations to ensure world class cell processing and preparation of quality cell and gene products. Our apheresis and transplant service were the first in NSW to be licenced by the TGA. We are also familiar with both U.S. Food & Drug Administration and European Medicines Agency requirements including PIC/s.

Process development

Cell manufacturing processes are taken from research protocol through to clinically relevant scale in a separate process development laboratory. Staff training, documented processes and material and equipment qualification ensure that the developed processes will enable the manufacture of safe and consistent products in the cGMP environment.


The self-contained, state of the art facility of over 400 m2 has been designed with flexibility to meet clinical research requirements. The manufacturing space includes four separate clean room suites, a large quality control and support laboratory, raw materials and cell product storage facilities plus scientific and administrative support areas. The facility can accommodate the diverse needs of clinical academic researchers, as well as international biotechnology industry partners.

We have two large positive pressure, and two smaller negative pressure clean rooms to accommodate conventional and gene modification protocols. Isolated entry and exit gantries for each laboratory ensure unidirectional personnel flow. A central support laboratory provides common storage space and Quality Control testing facilities.

All critical equipment used for product processing or storage is connected to a central environmental monitoring system that operates 24/7. Local visual alarms and remote alarms alert staff to ensure product quality and safety. The facility was designed to be compliant with the Australian TGA standards for the manufacture of human cells for therapeutic use and the Office of the Gene Technology Regulator for clinical manufacturing protocols involving genetic modification.

Key Achievements


Cell & Molecular Therapies is establishing a new program in the translation of cell and gene therapies. We work closely with local and international collaborators including the Children's Hospital of Philadelphia, and Athersys Inc. to develop new genetic and cell based therapies.

We are bringing ground-breaking treatment options to our patients through participation in an international phase I/II clinical trial of gene modified stem cells for the treatment of beta-thalassemia , as well in as a series of related Phase I/II trials using adeno-associated viral vectors for the treatment of haemophilia B or factor IX deficiency.

Cell & Molecular Therapies are now able to offer a new service to local clinicians; the preparation of autologous serum eye drops (ASED) for the treatment of dry eye syndrome. Patients attend for a simple blood draw and their blood is processed into a preparation that they can use to alleviate the symptoms of dry eye syndromes. In 2015 Cell & Molecular Therapies joined the Co-operative Research Centre for Cell Therapy Manufacturing, primarily based in Adelaide and continued as a member of the Therapeutic Innovation Australia initiative know as the Australian Therapeutic Pipeline.


Our research team continues to elucidate an entirely new mechanism by which genes are switched off in normal white blood cells that may lead to new therapeutic targets for cancer and leukaemia. The hidden mechanism was revealed through understanding a new function of the mysterious 'junk DNA' which makes up by far the majority of our genetic material. We realised that many genes use a 'molecular trash can' that is activated by genetic 'junk' called 'introns' to dispose of unwanted gene expression.

Cancer is caused by the accumulation of mutations (errors) in our DNA. Cancer causing mutations activate oncogenes or inactivate tumour suppressor genes. Multiple DNA mutations lead to the development of cancer. One tumour suppressor gene called CTCF is a DNA binding protein that is important for normal organisation of the chromatin, found in our chromosomes. Mutations and deletions of the CTCF gene occur in many cancer types including blood cancer. We are working to understand how CTCF functions in normal cells, and how changes in the CTCF gene lead to cancer development.

Cancer cells exhibit uncontrolled growth in the body and cellular nutrients must be imported into a cancer cell to sustain this growth. We have discovered that pumps responsible for nutrient uptake are increased in different cancer types. Our research is now determining ways to improve cancer therapies by blocking these nutrient pumps, thereby starving cancer cells.


Distinguished Professorial Achievement Award, Sydney Medical School, University of Sydney. In recognition of distinguished sustained achievements by professors in research, teaching and learning as well as service to Sydney Medical School, the University and the profession.

Research Staff

Cell & Molecular Therapies RPAH

  • Professor John Rasko, Director, Head of Department
  • Dr Stephen Larsen, Staff Specialist, Institute of Haematology, RPAH
  • Dr Janet Macpherson, Development Manager
  • Mr Angel Jaramillo, Production Manager
  • Dr Zlatibor Velickovic, Quality Officer
  • Dr Michelle Keir, Project Manager
  • Ms Natasha Barry, Hospital Scientist
  • Ms Flora Kan, Hospital Scientist
  • Ms Afroditi Sdrolias, Hospital Scientist
  • Ms Gemma Williamson, Hospital Scientist
  • Mr James Favaloro, Hospital Scientist
  • Ms Linda Pallot, Registered Nurse

Gene and Stem Cell Therapy Group CENTENARY

  • John EJ Rasko, Head
  • Jeff Holst, Associate Faculty
  • William Ritchie, Associate Faculty
  • Chuck Bailey, Senior Research Officer
  • Amy Marshall, Research Officer
  • Justin Wong, Research Officer
  • Kevin Wang, Research Officer
  • Katherine Lau, Research Officer
  • Carl Power, Editorial Research Officer
  • Cynthia Metierre, Research Assistant
  • Katherine Champ, Research Assistant
  • Natalia Pinello, Research Assistant
  • Yue Feng, Research Assistant
  • Rajini Nagarajah, Research Assistant

Higher Degree Students:


  • Ms Keren Weiss
  • Mr Kareishma Kabani
  • Ms Fiona Guan
  • Dr Jane Gordon
  • Dr Teh Liane Khoo

Publications and Presentations

Publication Details:
  1. Wang Q, Beaumont KA, Otte NJ, Font J, Bailey CG, van Geldermalsen M, Sharp DM, Tiffen JC, Ryan RM, Jormakka M, Haass NK, Rasko JEJ, Holst J. Targeting glutamine transport to suppress melanoma cell growth. International Journal of Cancer, Sept 2014; 135(5):1060-1071 IF:6.198 Cit:16
  2. Wong J -L, Lau K, Pinello N, Rasko JEJ. Epigenetic modifications of splicing factor genes inmyelodysplastic syndromes and acute myeloid leukemia. Cancer Science, Nov 2014; 105(11):1457-1463 IF: 3.534 Cit:4
  3. Clancy JL, Patel HR, Hussein SMI, Tonge PD, Cloonan N, Corso AJ, Li M, Lee DS, Shin JY, Wong JJL, Bailey CG, Benevento M, Munoz J, Chuah A, Wood D, Rasko JEJ, Heck AJR, Grimmond SM, Rogers IM, Seo JS, Wells CA, Puri MC, Nagy A & Preiss T. Small RNA changes en route to distinct cellular states of induced pluripotency. Nature Communications, Dec 2014; 10(5):5522 IF:10.742 Cit:14
  4. Hussein SMI, Puri MC, Tonge PD,Benevento M, Corso AJ, Clancy JL, Mosbergen R, Li M, Lee DS, Cloonan N, Wood DL, Munoz J, Middleton R, Korn O, Patel HR, White CA, Shin JY, Gautheir ME, Le Cao, KA, Kim JL,Mar JC, Shakiba N, Ritchie W, Rasko JEJ, Grimmond SM, Zandstra PW, Wells CA, Preiss T, Rogers IM, Seo JS, Heck AJR, Nagy A. Genome-wide characterization of the routes to pluripotency. Nature, Dec 2014; 516(7530):198-206 IF: 42.324 Cit:41
  5. Wang Q, Hardie RA, Hoy AJ, van Geldermalsen M, Gao D, Fazli L, Sadowski MC, Balaban S, Schreuder M, Nagarajah R, Wong JJ, Metierre C, Pinello N, Otte NJ, Lehman ML, Gleave M, Nelson CC, Bailey CG, Ritchie W, Rasko JEJ, Holst J. Targeting ASCT2-mediated glutamine uptake blocks prostate cancer growth and tumour development. Journal of Pathology, Jul 2015; 236(3):278-289 IF 2.62 Cit:1

  1. Lui H-J, Ooms LM, Srijakotre N, Vieusseux J, Chai R, Waters JAE, Bailey CG, Rasko JEJ, Price JT, Mitchell CA. Molecular mechanisms by which P-Rex1 acts as an oncogene in breast cancer. 2014 International Association for Breast Cancer Research, Novotel Sydney, Manly Pacific, Australia, 14-17 September 2014
  2. Van Geldermalsen M, Wang Q, Bailey CG, Fen Y, Nagarajah R, Rasko JEJ, Holst J. Targeting the ASCT2 glutamine uptake pathway suppresses cell growth in triple-negative breast cancer. 2014 International Association for Breast Cancer Research, Novotel Sydney, Manly Pacific, Australia, 14-17 September 2014
  3. Leboulch P, and the LentiGlobin clinical trial study group (by alphabetic order): Beuzard Y, Cavazzana M, Fucharoen S, Hacein-Bay Abina S, Ho J, Hongeng S, Kwiatkowski JL, Larsen S, Macpherson J, Negre O, Payen E, Petrusich A, Rasko JEJ, Schiller G, Soni S, Thompson AA, von Kalle C, Walters M. From bench to bedside: gene therapy for the beta-hemoglobinopathies. 9th Australiasian Gene & Cell Therapy Society (AGCTS) Conference, University of Melbourne, VIC, Australia, 29 April-1May 2015
  4. Rasko JEJ, Ho J, Larsen S, Macpherson JL, Thompson AA, Walters M, Petrusich A, Soni S, Hongeng S, Kwiatkowski JL, Schiller G, f von Kalle C, Leboulch P, Cavazzana M. Down Under where? Overcoming the challenges of an international multicentre β-thalassemia major study of LentiGlobin BB305-gene-modified autologous CD34+ cells with centralised manufacturing. 21st ISCT Annual Meeting, Las Vegas, USA, 27-30 May 2015
  5. Dominici M, Nichols K, Srivastava A, Weiss DJ, Eldridge P, Cuende N, Deans RJ, Rasko JEJ, Levine AD, Turner L, Griffin DL, O'Donnell L, Forte M, Mason C, Wagena E, Janssen W, Nordon R, Wall D, Ho H-N, Ruiz MA, Wilton S, Horwitz EM & Gunter KC, all belonging to the 2013-2015 ISCT Presidential task force on unproven cellular therapy. Positioning a scientific community on unproven cellular therapies: The 2015 International Society for Cellular Therapy Prespective, Cytotherapy, Nov 2015
  6. Velickovic Z, Barry N, Keir MW, Sdrolias A, Kan F, Jaramillo A, Wright C, Macpherson JL, Larsen SR, Rasko JEJ. Designing and validating an environmental monitoring program. 21st ISCT Annual Meeting, Las Vegas, USA, 27-30 May 2015
  7. Edwards CR, Middleton R, An X, Mishra T, Mohandas N, Hardison R, Rasko JEJ, Blobel GA. A dynamic intron retention program in the mammalian megakaryocyte and erythrocyte lineages. 56th ASH Annual Meeting and Exposition, Orlando, Florida, USA, 5-8 December 2015
  8. Walters MC, Rasko JE, Hongeng S, Kwiatkowski J, Schiller GJ, Kletzel M, Ho JP, Vichinsky E von Kalle C, Cavazzana M, Leboulch P, Petrusich A, Soni S and Thompson AA. Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of
  9. Gene Therapy for Beta-Thalassemia Major Via Transplantation ofAutologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305 Drug Product). 56th ASH Annual Meeting and Exposition, Orlando, Florida, USA, 5-8 December 2015
  10. 'Stem cell research', 10th Annual Symposium, Medivision Menzies Research Institute, University of Tasmania, Hobart, 19-20 July 2014
  11. 'Orchestrated intron retention regulates normal granulocyte differentiation', EMBO, Polonia Castle Pultusk, Poland, 11-16 September 2014
  12. 'Cell and gene therapy; coming to terms with it all', 5th MTERMS, Hotel Bangi-Putrajaya, Malaysia, 17-19 September 2014
  13. 'Serendipity and Science; from Gene Therapy to Cancer via the Genetics of Aminoaciduria', 5th MTERMS, Hotel Bangi-Putrajaya, Malaysia, 17-19 September 2014
  14. 'Intron retention provides a hidden layer of gene expression control', Harry Perkins Institute of Medical Research, Perth, 23 October 2014
  15. 'Intron retention provides a hidden layer of gene expression control', Institute of Stem Cell Biology & Regenerative Medicine, Stanford University, Stanford CA USA, 11 December 2014
  16. 'SCADART: Driving progress in the iPSC space', 2nd Annual Phacilitate Cell & Gene Therapy Forum, Grand Hyatt, Washington DC, USA, 26-28 January 2015
  17. 'Intron retention is a conserved gene expression control mechanism associated with epigenetic regulation', Human Genome Meeting 2015, Kuala Lumpur Convention Centre, Malaysia, 14-17 March 2015
  18. 'CTCF and endometrial cancer - unravelling the weave, ANZGOG Annual Scientific Meeting, Sofitel Gold Coast, 25-28 March 2015
  19. 'Stem Cells Now and in the Future - Where are we on the road to translation?, ARCS Scientific Congress, Royal Randwick Turf Club, Sydney, 5-7 May 2015
  20. 'Clinical Challenges for NanoMedicine in Cell and Gene therapy', 6th International NanoMedicine Conference, Crowne Plaza, Coogee, 6- 8 July 2015
  21. 'Clinical reality of stem cell treatments', Forum on community expectation in stem cell science, Melbourne Brain Centre, Melbourne,10 September 2015
  22. 'A Personalised approach to patient care', RACP Convocation Ceremony, Melbourne Exhibition & Convention Centre, Melbourne 24 September 2015
  23. 'From Gene Expression to Genes as Medicine', AAHMS Annual Scientifics Meeting, The John Curtin School of Medical Research, Canberra, 6 October 2015
  24. 'Future directions in cancer biology and targeted therapeutics', 20th Weizmann Australia Symposium, Melbourne Museum, Melbourne 19 October 2015
  25. 'Current status and anticipated developments in cellular therapies', Australian and New Zealand Burn Association (ANZBA) Annual Scientific Meeting, Crown Convention Centre, Melbourne, 20 October 2015
  26. 'Blood, fret and tears?', Grand Rounds, RPAH, Scot Skirving Lecture Theatre, Camperdown, 23 October 2015
  27. 'Intron retention is an epigenetically regulated mechanism of gene expression control in normal biology & cancer. 6th Australian Epigenetic Conference, Hobart, 12-14 November 2015
  28. 'The Cell and Gene Therapy Soup: Disentangling hope, realism and quackery ',Stem Cell Society Singapore Symposium 2015, Singapore, 17-18 November 2015
  29. 'Regulation of Gene and Cell Therapy', Immunotherapy@Brisbane 2015, Brisbane Convention and Exhibition Centre, Brisbane, 24-26 November 2015
  30. 'Disentangling hope, realism and quackery in Cell and Gene therapy', Hematology Department in Santiago, Chile, 10 December 2015
  31. 'Intron retention provides a hidden layer of gene expression control', Illawarra Health and Medical Research Institute, University of Wollongong, 15 October 2014
  32. 'Getting something for nothing?, Intron retention downregulates gene expression' Lowy Cancer Research Centre, UNSW, Kensington Campus, Randwick, 5 November 2014
  33. 'Personalised medicine beyond biomarkers', The George Institute for Global Health, Sydney, 2 September 2015
  34. 'Making cell and gene therapies a reality', 2015 Sydney Catalyst International Translational Cancer Research Symposium Program, Dockside, Darling Harbour, 27 July 2015

Research Grants

Clive and Vera Ramaciotti Foundations Centenary Institute The Ramaciotti Centre for Human Systems Biology  $     1,000,000 2014-2016
National Health & Medical Research Council Rasko Intron retention regulation in granulopoiesis and leukaemia  $       565,280 2014-2016
National Health & Medical Research Council Ritchie/Rasko Investigating widespread regulation of gene expression through intron retention  $       354,549 2015-2017
National Health & Medical Research Council Winkler/Rasko Haematopoietic Stem Cell glycome regulates outcome of niche interactions  $       131,832 2015-2018
New South Wales Cancer Council Rasko Consequences of CTCF haploinsufficiency in endometrial carcinoma  $       360,000 2014-2016
New South Wales Cancer Council Rasko Consequences of CTCF mutation in acute lymphoblastic leukaemia  $       360,000 2014-2016
New South Wales Cancer Council Holst Starving Cancer Cells: Developing nutrient uptake inhibitors as prostate cancer therapeutics  $       360,000 2015-2017
Office of Mecial Research Rasko OHMR Genomics Pilot: Characterising novel mechanisms controlling aberrant splicing in cancer (ASIC)  $       136,350 2015
Anonymous Rasko  n/a  $     3,164,640 2013-2017
Tour de Cure Bailey Optimising response to treatment in Chronic Myeloid Leukaemia (CML)  $       400,000 2013-2015
University of Sydney SU Holst Nutrient Transporters: elucidating and targeting their functions in cancer  $       130,000 2015
Anonymous Rasko/Macpherson Cell & Molecular Therapies  $       402,000 2013-2017
Anonymous Rasko/Macpherson CRC for Cell Therapy Manufacturing  $       250,000 2015-2019

Contact details for department

Head of Department: Professor John E J Rasko

Department/Unit: Cell and Molecular Therapies
RPA Hospital
Missenden Road
Camperdown NSW 2050
Telephone: (02) 9515 4860
Facsimile: (02) 9515 4868
Web: https://www.slhd.nsw.gov.au/research/
Email: janet.macpherson@sswahs.nsw.gov.au